Grants and Contracts per year
Grants and Contracts Details
To assess the safety of systemic administration of a novel self complementary AAV vector (scAAV2/8-LP1-hFIXco) in adults with severe hemophilia B at up to three dose levels. We predict that the systemic administration of scAAV2/8-LP1-hFIXco vector pseudotyped with AAV8 capsid protein and encoding the human FIX (hFIX) gene will be safe with absence of persistent Grade III or greater dose limiting toxicity. Any Grade I-II toxicity is likely to be dose dependent and reversible. To test this hypothesis we have developed a comprehensive clinical monitoring plan that will detect and document any adverse events following gene transfer.
|Effective start/end date
|10/1/13 → 1/31/16
- St Jude Childrens Research Hospital: $38,308.00
Explore the research topics touched on by this project. These labels are generated based on the underlying awards/grants. Together they form a unique fingerprint.